IV-delivered nanocapsules to edit genes across the brain for Alzheimer’s
Brain-Wide Genome Editing Enabled by Intravenously Administered Non-Viral Nanovectors As a Potential Therapy for Alzheimer’s Disease
This work tests whether tiny, non-viral capsules given through a vein can carry gene-editing tools across the whole brain to help people with Alzheimer’s disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Wisconsin-Madison NIH-funded |
| Lab location | 1 site (Madison, United States) |
| Project ID | NIH-11457089 on NIH RePORTER |
What this research studies
As someone affected by Alzheimer’s, I want treatments that reach the whole brain without brain surgery. This project is creating glutathione-responsive silica nanocapsules that can be given intravenously and cross the blood-brain barrier to carry CRISPR gene editors. The team will test these nanocapsules in a mouse model that carries a human-like APP mutation to see if editing that gene can reduce Alzheimer-type changes. They will also check safety and how well the approach reaches different brain regions.
Who could benefit from this research
Good fit: People with Alzheimer’s disease—especially those whose condition involves mutations or changes in the amyloid precursor protein (APP)—would be the eventual candidates for therapies developed from this research.
Not a fit: Patients without Alzheimer’s, those with dementia from unrelated causes, or people in very late-stage Alzheimer’s may not benefit from this specific APP-targeting approach.
Why it matters
Potential benefit: If successful, this approach could provide a non-invasive, brain-wide gene therapy that slows or prevents Alzheimer’s-related brain damage.
How similar studies have performed: Viral gene therapies and localized brain injections have shown promise in animals, but systemic non-viral delivery of CRISPR across the whole brain is largely novel and unproven in humans.
Where this research is happening
Madison, United States
- University of Wisconsin-Madison — Madison, United States (Active)
Researchers
- Principal investigator: Gong, Shaoqin - — University of Wisconsin-Madison
- Study coordinator: Gong, Shaoqin -
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.